BrainStorm's ALS therapy wins orphan drug status from FDA

Haim Leibovits

The FDA can apply orphan drug status to drugs for disorders affecting up to 200,000 people in the U.S. if the drugs have exhibited significantly better results than existing treatments

Biotech startup BrainStorm Cell Therapeutics has achieved a major breakthrough with the U.S. Food and Drug Administration: TheMarker has learned that a treatment it is developing for amyotrophic lateral sclerosis (ALS ) based on stem cell therapy has been granted orphan drug status.

Orphan drugs are those developed for relatively small markets of patients. The FDA can apply this designation to drugs for disorders affecting up to 200,000 people in the U.S. if the drugs have exhibited significantly better results than existing treatments.

Orphan drug status provides the medicine with market exclusivity in the U.S. for seven years and may entitle it to financial incentives of up to $1.6 million to offset clinical trials. It also allows for accelerated regulatory testing in the third and decisive set of clinical trials toward final FDA approval.

BrainStorm, controlled by Chaim Lebovits and Jacky Ben-Zaken, currently trades at a market value of $28 million on the OTC Bulletin Board after having declined 9% over the past year. In the last month, however, the stock has soared 65% following the appointment of a new acting chief executive, Dr. Adrian Harel.

The company plans to issue shares in Israel and an anonymous Israeli investor was reported last week to have bought $250,000 of the stock at a 20% premium over its current share price. Mori Arkin, who sold his drug company Agis Industries to Perrigo in 2004, was also reported to be interested in investing, and BrainStorm officials have been taking the company on a road show to institutional investors in the U.S.

Stem cell therapy is considered the up-and-coming field in medicine and the greatest medical breakthrough since antibiotics. BrainStorm's distinction is its use of adult bone marrow stem cells rather than embryonic stem cells in its treatment for ALS, also known as Lou Gehrig's disease.

BrainStorm's therapies are based on research conducted by neurologist Prof. Eldad Melamed and cell biologist Prof. Daniel Offen. The target diseases are ALS and other degenerative neurological conditions such as Parkinson's, multiple sclerosis and spinal cord injuries.

Treatment involves isolating stem cells taken from the patient's own bone marrow and transforming them in cultures into nerve cells that are then reinjected into the patient. The treatment is expected to slow, perhaps even halt, the degenerative process.

Over the last two years, the company has focused on treating ALS, which has long been considered incurable. In October, it received Health Ministry clearance to begin clinical trials of its stem cell therapy on ALS patients, and testing is expected to begin in the next few weeks at Jerusalem's Hadassah University Hospital, Ein Karem.

Adrian Harel was also appointed chief operating officer of Brainstorm. Avi Israeli, chairman of the board at Brainstorm, said at the time of the appointment on January 27 that Harel has experience in drug development and manufacturing "in addition to building and managing highly successful biotechnology companies".